Cabaletta for the treatment of Spinocerebellar Ataxia Type 3 (SCA3)
SCA3, also known as Machado Joseph disease, is the most common disease among the cerebellar ataxias, which are a group of genetic diseases that are characterized by memory deficits, spasticity, difficulty with speech and swallowing, weakness in the arms, and other muscular disorders. Symptoms can begin in early adolescence and get worse over time. In severe cases SCA3 can lead to an early death in the fourth decade of life. SCA3 is incurable, and there is currently no approved therapeutic treatment for the disease.
SCA3 is caused by a mutation in the DNA that leads to the creation of a pathological protein – Ataxin 3. Ataxin 3 is unstable, and aggregates within the nerve cells and eventually leads to cell death.
Cabaletta was found to be effective, both as an anti-mutant protein aggregation agent and as an autophagy enhancer, in reducing protein aggregates and improving cell survival in several spinocerebellar ataxias, including SCA3 cells. Additional animal studies have shown that activation of autophagy may be beneficial in alleviating disease symptoms.
BioBlast is currently conducting a Phase 2 study for Cabaletta in Spinocerebellar Ataxia Type 3.